Synageva BioPharma Corp. (GEVA) announced that the U.S. FDA has granted Breakthrough Therapy designation to sebelipase alfa for the treatment of early onset lysosomal acid lipase deficiency, also known as Wolman disease. The FDA has also confirmed that late onset LAL Deficiency is "a serious and life threatening disease or condition" and that Breakthrough Therapy designation could be obtained for this aspect of the disease with additional clinical information, the company said.
The designation was based on data generated from clinical trials with sebelipase alfa in patients with early onset LAL Deficiency.
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